FDA reverses course on Regenxbio's gene therapy for Hunter syndrome
The FDA will reconsider approving Regenxbio’s gene therapy for Hunter syndrome, STAT reported , reversing a rejection issued just four months earlier. Hunter syndrome, or mucopolysaccharidosis type II, is a rare inherited disorder that causes progressive cognitive decline in children; unlike existing enzyme-replacement treatments, the gene therapy is designed to reach the brain.
The reversal is the latest in a string of FDA about-faces that have followed leadership turnover, including the departures of former commissioner Marty Makary and other officials in recent weeks. It echoes a separate decision allowing UniQure to resubmit a Huntington’s disease gene therapy the agency had earlier rejected and the former commissioner had publicly criticized. The pattern suggests changing regulatory judgment on novel gene therapies, which gives developers a second path to market but raises questions about consistency in how the agency weighs the same data.